AAV gene therapy in hemophilia: progress, challenges and what comes next

This VJHemOnc podcast explores the evolving role of gene therapy in hemophilia, focusing on the challenges of AAV-based approaches and the development of next-generation strategies. Radek Kaczmarek, PhD, Indiana University School of Medicine, Indianapolis, IN, discusses the limitations of AAV gene therapy in hemophilia A, including factor VIII engineering, durability, and liver toxicity. He also examines potential malignancy risks associated with AAV integration and the importance of long-term surveillance following treatment. Finally, Dr Kaczmarek discusses emerging non-AAV approaches, including lentiviral gene therapy, targeted gene insertion, and gene editing, and their potential to address some of the limitations of current gene therapy approaches.